News

Ra Pharmaceuticals is preparing to launch a Phase 3 clinical trial to assess the therapeutic potential of zilucoplan for the treatment of patients with generalized myasthenia gravis (gMG). This comes after the company’s completion of End-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA). The 12-week study — a single, pivotal,…

Robotic surgical removal of the thymus seems to be safe and effective and may reduce or eliminate the need for steroid therapy in myasthenia gravis (MG) patients older than 60 at onset. The study, “Results of robotic thymectomy performed in myasthenia gravis patients older than 60 years at…

Alexion Pharmaceuticals‘ Soliris (eculizumab) significantly reduces fatigue perceived by patients with generalized myasthenia gravis, a study finds. The study, “Eculizumab improves fatigue in refractory generalized myasthenia gravis,” was published in Quality of Life Research. Myasthenia gravis (MG) is an autoimmune disease caused…

For more than four decades, comedian Jerry Lewis hosted the MDA Labor Day Telethon on behalf of the Muscular Dystrophy Association. That annual event helped the MDA become the nation’s largest non-government source of funding for neuromuscular disease research, with more than $1.4 billion disbursed since its establishment 68…

A recent report described a rare case of a woman presenting with a sudden decrease in voice volume who was ultimately diagnosed and successfully treated for myasthenia gravis (MG). The case study, “Hypophonia as only presenting symptom in myasthenia gravis — a diagnostic dilemma in poor…

Soliris (eculizumab) may be the most effective and well-tolerated immunotherapy for patients with myasthenia gravis (MG), according to an analysis of multiple studies. The research, “Immunosuppressive and monoclonal antibody treatment for myasthenia gravis: A network meta‐analysis,” was published in the journal CNS Neuroscience…

Prograf (Tacrolimus) may be a beneficial treatment for people with myasthenia gravis (MG), helping reduce steroid use and disease scores, a joint analysis of several studies shows. The review, “Effectiveness and safety of tacrolimus therapy for myasthenia gravis: A single arm meta-analysis” was published in the…

Although rare, myasthenia gravis can develop in patients with systemic lupus erythematosus, according to a case study. The study, “Systemic Lupus Erythematosus and Myasthenia Gravis: A Rare Association,” was published in The Indian Journal of Nephrology. Systemic lupus erythematosus (SLE) and myasthenia gravis (MG) are two autoimmune…

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bath dissertation, she analyzed Brexit’s long-term impact…

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…